Project HERCULES will develop a disease level economic model and other tools to better demonstrate the real value of new treatments for DMD for Health Technology Assessments and reimbursement decisions.

Project HERCULES has already achieved real advances in our understanding of DMD. We are bringing together possibly the largest collection of clinical data on DMD to develop a better understanding of which aspects of the disease are most important to patients and how they might best inform an economic model. We interviewed people living with DMD to understand better what has the most impact on their quality of life and will be publishing this in 2019. We have identified key evidence gaps and commissioned a burden of illness study to address these in 2019.

This project is a multi-stakeholder collaboration with international applicability. By engaging with global patient organisations, we hope it will pave the way forward for more evidence generation to aid reimbursement decisions worldwide for DMD and other rare diseases.

We were delighted that, in November, Project Hercules was awarded the EURORDIS Black Pearl Award for Patient Engagement in recognition of its innovative approach to bringing patients, clinicians, industry, academics, HTA organisations and others together to address the challenges of assessing new treatments in DMD.

Project HERCULES will deliver:

A bespoke, validated, Quality of Life metric being developed by the University of Sheffield

A natural history model developed for bringing together the largest collection of clinical data in DMD for multiple registries and trials. This work is led by the University of Leicester

A burden of illness study that will better capture the true impact of DMD on patients and their families being developed by HCD Economics

A disease level economic model that will be produced by SourceHEOR

Together these tools will support companies taking new treatments through HTA and reimbursement processes and should lead to faster, better and more consistent decision making and improved access to medicines for patients.

The concept of Project HERCULES could also benefit other rare conditions struggling to develop strong evidence of the value of new treatments.

Emily Crossley, Co-founder of Duchenne UK said:

Project HERCULES has gone from an idea to a real working collaboration in just over a 12 months which is a huge achievement. Duchenne UK is thrilled to be leading such a ground breaking initiative and are grateful to our industry partners for seeing the value of Hercules and for working with us to address one of the major challenges of drug development. We would like to thank all the boys, men and their families who have taken part in some of the data generation for HERCULES, and all of our charity and academic partners who have helped drive this forward.

Josie Godfrey, Project Director for Project HERCULES said:

I am delighted to be working with such engaged patients, companies, academics, clinicians and HTA agencies on a project that will help ensure new treatments for DMD have the best possible tools and evidence to support access and reimbursement decisions.

Dr Elin Haf Davies, CEO of Aparito said:

Project HERCULES is an extremely exciting initiative that has all the ingredients to transform patient access to drugs through collaborative, cross-industry working. We’re delighted to offer regulatory support in ensuring the EMA/ HTA Qualification of this approach and the various elements it consists of.

Janis Clayton, VP & GM UK Region, PTC Therapeutics Limited said:

This highly ambitious and visionary project is committed to providing the materials needed to address the challenges of patient access to new therapies for Duchenne. It successfully brings together the knowledge and perspectives of patient organisations, industry and academia. PTC have supported the project from its initiation and it is wonderful to see the progress made to date, as well as the enthusiastic engagement from all parties for Phase 2. Many congratulations to Duchenne UK on this great achievement.

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

Most read

Duchenne affects approximately 1 in every 3,500 boys that are born but only around 1 in every 50 million girls. It may be rare, but it does happen. We have been speaking to Feriel, a 26 year old woman living with Duchenne muscular dystrophy. She has written us a short blog about her experiences with Duchenne from diagnosis to now.

Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

We are proud to share with you our first ever Impact Report, read about the very real impact we are having and the many things we have been working on over the past six years to end Duchenne.
Read more

We will always store your personal details securely. We’ll use them to provide the service that you have requested, and communicate with you in the way(s) that you have agreed to. Your data may also be used for analysis purposes, to help us provide the best service possible. For full details see our Privacy Policy or contact us on [email protected]